Gene Therapy for CF using Cationic Liposome Mediated Gene Transfer: Phase I Trial 
nasal potential difference measurement. Tearing and sneezing are also possible 
at the time the gene is given or otherwise studied. It is possible, but very 
unlikely, that inflammation, pain, change in sense of smell, or other damage to 
a small area of tissue within the nose could be permanent. However, it is much 
more likely that any symptom of pain or inflammation which develops would 
resemble a "cold" or "sinus infection" and would be temporary (for example, 
symptoms might include fever, runny nose or cough). The discomforts listed 
above will be minimized using numbing medications or other measures whenever 
possible. 
c) Administration of the normal CF gene will require that I lie still on my side for 
approximately 60 minutes without moving my head, and lying on my side in bed 
for approximately four (4) hours. 
d) The scrapings and biopsies of my nose can be painful, and may be associated 
with a small amount of bleeding. Healing of the nasal biopsy requires approxi- 
mately two weeks and can be associated with pain, itching, runny nose, and/or 
minor bleeding. The risk of infection at the site of the biopsy is very small. 
e) One chest x-ray and two sinus x-rays will be performed as part of the study. The 
chances of developing cancer or other complications from this amount of radiation 
is very small, although the actual risks in an individual CF patient who has 
received multiple x-rays in the past as part of usual care is not known. 
SPECIAL RISKS AND DISCOMFORTS RELATED 
TO BEING PART OF A STUDY OF GENE ADMINISTRATION 
Because CF gene transfer is experimental, the risks associated with this procedure are 
not known. Several risks are possible: 
1. Administration of the normal CF gene using lipid could cause an allergic or 
inflammatory response which would make subsequent administration more 
difficult, or cause a significant allergic reaction to the lipid or the normal CF 
gene. This is very unlikely since lipid administration of the type planned here 
does not generally cause pronounced allergic or inflammatory reactions (for 
example, it has not caused substantial inflammation in studies in laboratory 
animals). In addition, the lungs of CF patients are already exposed to consider- 
able amounts of other genes (in the form of DNA already present in the airways) 
so that administration of an additional gene is unlikely to provoke a significantly 
enhanced allergic response. However, the allergic reaction which might be caused 
in a given patient after gene administration is not known. 
2. It is possible that the normal CF gene could enter cells other than those within the 
nose at the time of administration. For example, some blood cells could carry the 
CF gene to other parts of the body. In all gene transfer studies, a concern is that 
a new gene might enter a patient’s sperm or egg. However, multiple barriers 
within the nose should make it very unlikely that significant amounts of lipid or 
DNA would be delivered to other areas of the body. 
Recombinant DNA Research, Volume 18 
[849] 
